Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) and Alexion Pharma International Sarl today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved the Company’s New Drug Application (NDA) for the use of Soliris® (eculizumab) as a treatment for patients in Japan with paroxysmal nocturnal hemoglobinuria (PNH). PNH is an ultra-rare, debilitating and life-threatening blood disorder defined by chronic red blood cell destruction, or hemolysis. Soliris, a first-in-class terminal complement inhibitor, is the first therapy approved in Japan for the treatment of patients with PNH. Soliris received orphan drug designation from the MHLW in 2009 and was approved under the Ministry’s priority review process.
“The speedy approval of the Soliris NDA in Japan underscores the severity of PNH in Japanese patients and the significant clinical impact that this treatment provides to patients living with PNH,” said Leonard Bell, M.D., Chief Executive Officer of Alexion. “This regulatory approval marks another important step in our global commitment to the objective of providing access to Soliris to all patients who can benefit from it. We now look forward to working closely with the healthcare authorities in Japan to make Soliris available to patients as rapidly as possible.”
Soliris was approved as a treatment for patients with PNH by the U.S. Food and Drug Administration and the European Commission in 2007, and has since received similar approvals from the healthcare authorities in other countries, including Australia, Korea and Canada. Governments and private insurance companies have recognized the breakthrough innovation of Soliris and are now providing patients with broad access to Soliris in the United States, the largest European nations, and additional countries around the world. With the approval of the NDA, Alexion is working with the MHLW to facilitate patient access to Soliris. The Company continues to anticipate a commercial launch of Soliris in Japan by the end of 2010.
“PNH is very rare, but has a devastating impact on many of the patients that it affects. After decades of ground-breaking basic scientific research in Japan regarding PNH, it is particularly noteworthy that clinical studies, including the AEGIS trial in Japan, have shown that Soliris markedly reduces hemolysis, the underlying cause of the serious illness and shortened life-span associated with PNH,” said Yuzuru Kanakura, M.D., Ph.D., Professor of Hematology and Oncology at Osaka University Hospital in Suita, Japan, and lead investigator of the AEGIS study. “Japanese patients with PNH will soon have access to the same life-changing medical benefits of Soliris that are available to patients in other nations.”
The AEGIS Study in Japan
Alexion’s NDA for Soliris included data from AEGIS, an open-label registration study examining Soliris as a treatment for Japanese patients with PNH, (1) as well as data from the previously reported SHEPHERD and TRIUMPH (2,3) PNH registration trials, which were conducted in North America, Europe, and Australia. AEGIS was conducted during 2008 and included 29 patients at nine institutions throughout Japan.
In December 2008, Alexion reported positive results from AEGIS. (4) The prespecified primary efficacy endpoint of change in hemolysis was achieved with an 86 percent reduction (P